Clinical Trial

Disease: Relapsed or Refractory T-Cell Lymphoma, (NCT04502446)

Disease info:

Lymphoma is a type of blood cancer that develops when white blood cells called lymphocytes grow out of control. Lymphocytes are part of our immune system. They travel around our body in our lymphatic system, helping us fight infections. There are two types of lymphocyte: T lymphocytes (T cells) and B lymphocytes (B cells).

Lymphomas can be grouped as Hodgkin lymphomas or non-Hodgkin lymphomas, depending on the types of cell they contain. T-cell lymphomas are non-Hodgkin lymphomas that develop from T lymphocytes.

Most T-cell lymphomas develop from mature T cells. They usually affect older adults, typically people in their mid-60s. They are more common in men than in women. Occasionally, T-cell lymphoma can develop from immature T cells. This is known as T-cell lymphoblastic lymphoma. It tends to affect children and young adults.

In most cases, it is not known what causes T-cell lymphomas. In a few types of T-cell lymphoma, research has shown that certain viral infections or medical conditions can increase the risk of developing lymphoma. 

Human T-lymphotropic virus type 1 (HTLV-1) is linked with development of adult T-cell leukaemia/lymphoma (ATL).
Past infection with Epstein-Barr virus (EBV) is linked to the development of a range of lymphomas, including angioimmunoblastic T-cell lymphoma (AITL).
Enteropathy-associated T-cell lymphoma (EATL) is linked with coeliac disease.

Relapsed refers to when a patient has received active treatment, went off treatment and then the disease came back, whereas refractory refers to disease that is progressing despite active treatment.

T-cell lymphomas are rare. They account for around 1 in 10 cases of non-Hodgkin lymphoma. Around 700 people are diagnosed with T-cell lymphomas in the UK each year.
Official title:
A Phase 1, Open-Label, Multicenter, Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Anti-CD70 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX130) in Subjects With Relapsed or Refractory T or B Cell Malignancies

Study Director: Alissa Keegan, PhD, CRISPR Therapeutics



Phone: +1 (877) 214-4634



United States, California

Research Site 2, Duarte, California, United States, 91010

Research Site 5, Stanford, California, United States, 94305


United States, Connecticut

Research Site 10, New Haven, Connecticut, United States, 06520


United States, Florida

Research Site 4, Miami, Florida, United States, 33124


United States, New York

Research Site 8, Bronx, New York, United States, 10467

Research Site 9, New York, New York, United States, 10065


United States, Texas

Research Site 1, Houston, Texas, United States, 77030


United States, Utah

Research Site 6, Salt Lake City, Utah, United States, 84112


Australia, New South Wales

Research Site 3, Sydney, New South Wales, Australia, 2050Canada, OntarioResearch Site 7RecruitingToronto, Ontario, Canada, M5G 2C1

Study start:
Jul. 31, 2020
45 participants
Gene editing method:
Type of edit:
Gene insertion, gene knock-out
anti-CD70 CAR, Major Histocompatibility Complex Class I (MHCI) , T Cell Receptor (TCR)
Delivery method:
Adeno-associated virus vector (AAV) - Ex-vivo
CTX130 allogeneic CAR-T cells targets CD70, an antigen expressed on both hematologic cancers, including certain lymphomas, and solid tumors, including renal cell carcinoma.
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


This is a single-arm, open-label, multicenter, Phase 1 study evaluating the safety and efficacy of CTX130 in subjects with relapsed or refractory T or B cell malignancies.

CTX130 CD70-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components. Three modifications to healthy donor T cells are made to allow CAR-T cells to be used off-the-shelf:

CAR: The chimeric antigen receptor (CAR) allows CAR-T cells to target and kill cancer cells. A CAR has two key domains: one that binds to the surface of cancer cells and another that activates the T cell. In CTX130 CRISPR-Cas9 is uded to insert the CAR construct precisely into the TCR alpha constant (TRAC) locus, which it is expected to result in a safer, more consistent product.

TCR: T cells use the T cell receptor (TCR) to recognize and kill cells presenting foreign antigens (a sign of infection), thereby providing immunity from disease. Donor T cells could also recognize a patient’s cells as foreign through this receptor, leading to an unwanted side effect known as graft versus host disease (GvHD). CTX130 uses CRISPR-Cas9 to eliminate the TCR with high efficiency, which reduces the risk of GvHD occurring during off-the-shelf use.

MHC I: To improve CAR-T cell persistence and increase the chance for durable remissions, CTX130 uses CRISPR-Cas9 to eliminate the class I major histocompatibility complex (MHC I) expressed on the surface of the CAR-T product candidate. If present, MHC I could lead to rejection of the CAR-T product by the patient’s own T cells. Eliminating this molecule should mitigate that effect.

Last updated: Dec. 28, 2023
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